Observational Study on the Use of Ropeginterferon Alfa-2b in Polycythemia Vera (ROPEG-PV)

July 8, 2026 updated by: FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS

Polycythaemia vera (PV) is associated with a reduced quality of life, a high rate of vascular events, and an intrinsic risk of disease evolution. The results of several randomised trials for the treatment with new cytoreductive agents are now available, among which a new ropegylated formulation of interferon alfa-2b (ropeginterferon alfa-2b) have been recently approved in Europe and USA [EMA (2019), FDA (2021) and AIFA (2022)]. The use of this drug in clinical practice is an opportunity for a prospective observational study in a rare disease such as PV; the aim is to evaluate its impact in the practical management of these patients.

Therefore, the main objectives of the present study are to determine:

(i) to what extent ropeginterferon alfa-2b can be prescribed and tolerated in patients with PV; (ii) the risk-benefit of ropeginterferon alfa-2b in patients with PV, followed-up in real-world clinical practice.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Classical Philadelphia-negative myeloproliferative neoplasms (Ph-neg MPNs) including polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF) are characterized by uncontrolled clonal proliferation of multipotent bone marrow progenitors, sustained by acquired mutations in JAK2, CALR and MPL genes.

Natural history of PV is marked by life threatening outcomes such as thrombosis, bleeding and clonal evolution towards myelofibrosis and acute myeloid leukemia. Treatment-relevant risk stratification is designed to estimate the likelihood of thrombotic complications, which is estimated to occur before or after diagnosis in 20-30% of patients according disease and patient-related risk factors. The cornerstone of treatment in PV includes scheduled phlebotomy, with a hematocrit (Hct) target of <45% and low-dose aspirin in all patients, regardless of risk category. There is currently broad consensus regarding the need for cytoreductive drugs in high-risk patients with PV identified by age >60 years and prior history of thrombosis.

The results of several andomized trials for the treatment of PV are now available, and, in addition to the standard drug hydroxyurea (HU), both a new ropegylated formulation of interferon alfa-2b3 and ruxolitinib4 are now available have been approved in Europe and US and European LeukemiaNet (ELN) investigators have recently provided recommendations for the use of these drugs in clinical practice in low-risk as well as high-risk patients.

After approval by EMA (2019) and FDA (2021), the drug (ROPEGINTERFERON ALFA-2B) was very recently approved and reimbursed by AIFA (2022) in some subgroups of patients with PV. The use of this drug in clinical practice is an opportunity for a prospective observational study in a rare disease such as PV; the aim is to evaluate its impact in the practical management of these patients, according to Determinazione AIFA 20 marzo 2008 about observational clinical studies, and Decreto Ministeriale 17 dicembre 2004 on non-profit studies.

It is not entirely known which is the percentage of patients who, after careful screening as required in good clinical practice, will fail the indications for concomitant clinical or laboratory abnormalities. Furthermore, the proportion of patients who discontinue the drug during follow-up for intolerance or other reasons is currently unknown and data on the benefit-risk ratio are limited.

Moreover, it should be noted that the haematological and clinical responses obtained in clinical trials not always are replicated in the studies of the real-world clinical practice. In fact, daily management of PV patients does not require the same stringent enrollment and follow-up criteria as instead are necessary in clinical trials. Our proposal may also contribute to better implement the results following the recent guidelines, particularly in some subgroups of patients in which AIFA has established the use with reimbursement by Italian National Health System (NHS) (i.e., patients intolerant to HU, women of childbearing age who plan pregnancy and patients with history of skin cancer).

Study Type

Observational

Enrollment (Estimated)

429

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Italy
      • Alessandria, Italy, 15121
        • Recruiting
        • A.S.O. SS. Antonio e Biagio e C.Arrigo di Alessandria
        • Principal Investigator:
          • Monia Marchetti
        • Contact:
      • Bari, Italy
        • Recruiting
        • Azienda Ospedaliera Universitaria Consorziale - Policlinico, U.O. Ematologia con Trapianto
        • Contact:
        • Principal Investigator:
          • Alessandra Ricco, MD
      • Bologna, Italy
        • Recruiting
        • Policlinico S. Orsola - Malpighi, Unità di Ematologia
        • Principal Investigator:
          • Francesca Palandri, MD
      • Brescia, Italy, 25123
        • Recruiting
        • ASST-Spedali Civili
        • Contact:
        • Principal Investigator:
          • Rosa Daffini
      • Cagliari, Italy
        • Recruiting
        • Ospedale Businco, S.C. Ematologia e CTMO
        • Contact:
        • Principal Investigator:
          • Olga Mulas, MD
      • Catania, Italy
        • Recruiting
        • Azienda Ospedaliero - Universitaria "Policlinico Vittorio Emanuele" - PO Gaspare Rodolico, Dipartimento di Ematologia con Trapianto di midollo Osseo
        • Principal Investigator:
          • Giuseppe Palumbo, MD
        • Contact:
        • Contact:
        • Sub-Investigator:
          • Emma Cacciola, MD
        • Sub-Investigator:
          • Rossella Cacciola, MD
      • Cuneo, Italy, 2100
        • Recruiting
        • Azienda Ospedaliera S. Croce e Carle di Cuneo- Divisione di Ematologia,
        • Contact:
        • Principal Investigator:
          • Davide Rapezzi, Dr
      • Ferrara, Italy
        • Recruiting
        • Arcispedale Sant'Anna Azienda Ospedaliero - Universitaria di Ferrara, Unità Operativa di Ematologia
        • Principal Investigator:
          • Antonio Cuneo, MD
        • Contact:
        • Contact:
        • Sub-Investigator:
          • Giuseppe Auteri, MD
      • Florence, Italy
        • Recruiting
        • Azienda Ospedaliera Universitaria Careggi, Divisione di Ematologia
        • Contact:
        • Principal Investigator:
          • Alessandro Maria Vannucchi, MD
      • Messina, Italy
        • Recruiting
        • Azienda Ospedaliera Universitaria Policlinico "G. Martino", UOC Ematologia
        • Principal Investigator:
          • Alessandro Allegra, MD
        • Contact:
      • Mestre, Italy
        • Recruiting
        • Ospedale dell'Angelo, Dipartimento di Ematologia
        • Contact:
        • Principal Investigator:
          • Roberta Rocconi, MD
      • Milan, Italy
        • Recruiting
        • Ospedale San Raffaele, Unità Operativa di Ematologia e Trapianto Midollo Osseo
        • Contact:
        • Principal Investigator:
          • Daniele Sannipoli, MD
      • Milan, Italy
        • Recruiting
        • Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Ematologia
        • Contact:
        • Principal Investigator:
          • Alessandra Iurlo, MD
      • Monza, Italy, 20900
        • Recruiting
        • ASST MONZA Ospedale San Gerardo Clinica Ematologica
        • Contact:
        • Principal Investigator:
          • Elena Maria Elli, Dr
      • Naples, Italy, 80131
        • Recruiting
        • Azienda Ospedaliera Universitaria Federico II di Napoli Divisione di Ematologia e Trapianti del Midollo
        • Contact:
        • Principal Investigator:
          • Fabrizio Pane, Prof
        • Sub-Investigator:
          • Novella Pugliese, Dr
      • Novara, Italy, 28100
        • Recruiting
        • Azienda Ospedaliero Universitaria Maggiore della Carità di Novara SCDU Ematologia
        • Contact:
        • Principal Investigator:
          • Andrea Patriarca, Dr
      • Palermo, Italy
        • Recruiting
        • Azienda Ospedaliera Universitaria Policlinico "P. Giaccone", Divisione di Ematologia
        • Contact:
        • Principal Investigator:
          • Marco Santoro, MD
      • Pisa, Italy
        • Recruiting
        • Azienda Ospedaliera Universitaria Pisana
        • Principal Investigator:
          • Sara Galimberti, MD
        • Contact:
      • Roma, Italy
        • Recruiting
        • Azienda Ospedaliera San Eugenio - UOC Ematologia
        • Principal Investigator:
          • Elisabetta Abruzzese, MD
        • Contact:
      • Roma, Italy
      • Roma, Italy
        • Recruiting
        • Policlinico Umberto I, Dipartimento Ematologia, Oncologia e Dermatologia
        • Contact:
        • Contact:
        • Principal Investigator:
          • Massimo Breccia, MD
      • San Giovanni Rotondo, Italy
        • Recruiting
        • Ospedale Casa Sollievo della Sofferenza Istituto di Ricovero e Cura a Carattere Scientifico, U.O. Ematologia
        • Contact:
        • Principal Investigator:
          • Angelo Michele Carrella, MD
      • Torino, Italy, 10126
        • Recruiting
        • A.O.U. Città della Salute e della Scienza di Torino - Ospedale Molinette- S.C. Ematologia U
        • Contact:
        • Principal Investigator:
          • Giulia Benevolo, Dr
      • Trieste, Italy
      • Udine, Italy
        • Recruiting
        • Azienda Sanitaria Universitaria Integrata, Presidio Ospedaliero "Santa Maria della Misericordia", Clinica Ematologica
        • Contact:
        • Principal Investigator:
          • Mario Tiribelli, MD
    • Lombardy
      • Bergamo, Lombardy, Italy, 24127
        • Recruiting
        • SC Ematologia, ASST Papa Giovanni XXIII
        • Contact:
        • Sub-Investigator:
          • Annalisa Condorelli, MD
        • Principal Investigator:
          • Federico Lussana, MD
      • Milan, Lombardy, Italy, 20162
        • Recruiting
        • Divisione Ematologia ASST, Grande Ospedale Metropolitano Niguarda
        • Contact:
        • Principal Investigator:
          • Marianna Caramella, MD
      • Pavia, Lombardy, Italy, 27100
        • Recruiting
        • Divisione Ematologia, Fondazione IRCCS Policlinico San Matteo
        • Contact:
        • Principal Investigator:
          • Elisa Rumi, MD
      • Varese, Lombardy, Italy, 21100
        • Recruiting
        • U.O. Ematologia, Ospedale di Circolo e Fondazione Macchi Varese
        • Contact:
        • Principal Investigator:
          • Margherita Maffioli, MD
    • Veneto
      • Padova, Veneto, Italy, 35128
        • Recruiting
        • Clinica Medica I Azienda Ospedaliera di Padova
        • Contact:
        • Principal Investigator:
          • Irene Bertozzi, MD
      • Verona, Veneto, Italy, 37134
      • Vicenza, Veneto, Italy
  • Poland
      • Gdansk, Poland
        • Recruiting
        • University Medical Center, Department of Hematology and Transplantation
        • Contact:
          • Marta Sobas
        • Principal Investigator:
          • Maria Bieniaszewska, MD
      • Katowice, Poland
        • Recruiting
        • Pratia Onkologia, Department of Hematology and Cancer Prevention
        • Principal Investigator:
          • Sebastian Grosicki, MD
        • Contact:
          • Marta Sobas
      • Krakow, Poland
        • Recruiting
        • Jagiellonian University Hospital, Department of Haematology
        • Contact:
          • Marta Sobas
        • Principal Investigator:
          • Tomasz Sasha, MD
      • Lodz, Poland
        • Recruiting
        • Copernicus Hospital
        • Contact:
          • Marta Sobas
        • Principal Investigator:
          • Joanna Gora-Tybor, MD
      • Wroclaw, Poland
        • Recruiting
        • Medical University, Clinical Department of Haematology, Blood Neoplasms and Bone Marrow Transplantation
        • Principal Investigator:
          • Marta Sobas, MD
        • Contact:
          • Marta Sobas

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Sampling Method

Non-Probability Sample

Study Population

The first time point to assess the primary outcome defined as the achievement of CHR is after 12 months of treatment with ropeginterferon alfa-2b, is reasonable to provide evidence of at least a non-inferiority in terms of CHR with this new drug. In this framework, a sample size is calculated to test 1-sample non-inferiority of a proportion. Setting the expected proportion p of CHR under ropeginterferon alfa-2b equals to the reference value (p0) obtained with ruxolitinib (i.e., p = p0 = 0.24), a sample size of 319 is needed to demonstrate that the proportion of CHR under ropeginterferon alfa-2b is not inferior to that obtained under ruxolitinib, allowing a margin of non-inferiority (delta) of 0.07 and assuming alpha and beta errors of 5% and 10%, respectively.

Description

Inclusion Criteria:

  • Patients diagnosed with Polycythemia Vera by WHO 2016
  • Patient aged ≥ 18 years old
  • Patients in need of cytoreductive treatments with ropeginterferon alfa-2b in first or later lines according to the reimbursability criteria defined by the Italian National Health System
  • Patients who have signed the written informed consent for study participation.

Exclusion Criteria:

• Any contraindication for ropeginterferon alfa-2b according to the SmPC

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete hematological remission (CHR)* after 1 and 2 years of treatment.
Time Frame: At baseline and during the follow up at 6/12/18/24 months per patient.
*HCT lower than 45% without phlebotomies in the past 3 months; platelet count ≤ 400x109/L, WBC count <10 x109/L
At baseline and during the follow up at 6/12/18/24 months per patient.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete hematological (CHR) and clinical remission (CR) after 1 and 2 years of treatment stratified by different eligibility categories for treatment indication.
Time Frame: At baseline and during the follow up at 6/12/18/24 months per patient.
Secondary Outcomes for efficacy
At baseline and during the follow up at 6/12/18/24 months per patient.
Dose-response effect on CHR and CR
Time Frame: At baseline and during the follow up at 6/12/18/24 months per patient.
Secondary Outcomes for efficacy
At baseline and during the follow up at 6/12/18/24 months per patient.
Frequency of phlebotomies
Time Frame: At baseline and during the follow up at 6/12/18/24 months per patient.
Secondary Outcomes for efficacy
At baseline and during the follow up at 6/12/18/24 months per patient.
Incidence of any of the following: o arterial and venous thrombotic events; o hemorrhagic events; o disease related symptoms; o disease evolutions into myelofibrosis and acute leukemia; o secondary malignancies.
Time Frame: At baseline and during the follow up at 6/12/18/24 months per patient.
Secondary Outcomes for efficacy
At baseline and during the follow up at 6/12/18/24 months per patient.
Incidence, causality and intensity of any adverse event occurring during study period (as defined by MedDRA code and graded according to Common Terminology Criteria for Adverse Events (CTCAE last version)).
Time Frame: At baseline and during the follow up at 6/12/18/24 months per patient.
Secondary Outcomes for safety
At baseline and during the follow up at 6/12/18/24 months per patient.
Screening failure
Time Frame: At baseline and during the follow up at 6/12/18/24 months per patient.
Percentage of patients who, after careful screening as required in good clinical practice, will fail the indications for concomitant clinical or laboratory abnormalities.
At baseline and during the follow up at 6/12/18/24 months per patient.
Change in spleen size
Time Frame: At baseline and during the follow up at 6/12/18/24 months per patient.
Change in spleen size (from baseline) evaluated by palpation.
At baseline and during the follow up at 6/12/18/24 months per patient.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS

Investigators

  • Study Director: TIZIANO BARBUI, MD, FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 8, 2022

Primary Completion (Estimated)

September 30, 2026

Study Completion (Estimated)

December 31, 2029

Study Registration Dates

First Submitted

July 11, 2024

First Submitted That Met QC Criteria

July 11, 2024

First Posted (Actual)

July 17, 2024

Study Record Updates

Last Update Posted (Actual)

July 9, 2026

Last Update Submitted That Met QC Criteria

July 8, 2026

Last Verified

July 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ROPEG-PV

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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